Add Yahoo as a preferred source to see more of our stories on Google. FILE PHOTO: A Pfizer logo is displayed at a research facility in the La Jolla neighborhood of San Diego, California, U.S., ...
The hold followed Intellia’s voluntary pause in response to a grade 4 liver event—a case of Hy’s Law—in the ATTR-CM trial.
After a spate of patient deaths in 2025 linked to the company’s Duchenne gene therapy, Sarepta shared new data showing benefits of the therapy three years after dosing.
A five-year-old patient dosed with RGX-111 has developed a brain tumour four years after being dosed with the gene therapy.